Novel Translational Read-through–Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome

Shwachman-Diamond syndrome (SDS) is one of the most commonly inherited bone marrow failure syndromes (IBMFS). In SDS, bone marrow is hypocellular, with marked neutropenia. Moreover, SDS patients have a high risk of developing myelodysplastic syndrome (MDS), which in turn increases the risk of acute...

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Detalles Bibliográficos
Autores principales: Bezzerri, Valentino, Lentini, Laura, Api, Martina, Busilacchi, Elena Marinelli, Cavalieri, Vincenzo, Pomilio, Antonella, Diomede, Francesca, Pegoraro, Anna, Cesaro, Simone, Poloni, Antonella, Pace, Andrea, Trubiani, Oriana, Lippi, Giuseppe, Pibiri, Ivana, Cipolli, Marco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9024944/
https://www.ncbi.nlm.nih.gov/pubmed/35453634
http://dx.doi.org/10.3390/biomedicines10040886

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9024944/
https://www.ncbi.nlm.nih.gov/pubmed/35453634
http://dx.doi.org/10.3390/biomedicines10040886

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